Table of genes tested by the next-generation sequencing panel. (PPTX 58.1 kb

Brandon Hayes-Lattin

Elie Traer

Jeffrey W. Tyner

Jennifer Dunlap

Jessica T. Leonard

Philipp W. Raess

Journal of Hematology & Oncology

Table of genes targeted by siRNA panel in Fig. 2. (PPTX 47.3 kb

Jessica Leonard (3468890)

Philipp Raess (3468899)

Jennifer Dunlap (3468896)

Brandon Hayes-Lattin (3468902)

Jeffrey Tyner (3468893)

Elie Traer (755265)

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Additional file 3: Table S2. of Functional and genetic screening of acute myeloid leukemia associated with mediastinal germ cell tumor identifies MEK inhibitor as an active clinical agent

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Functional and genetic screening of acute myeloid leukemia associated with mediastinal germ cell tumor identifies MEK inhibitor as an active clinical agent

Additional file 2: Table S1. of Functional and genetic screening of acute myeloid leukemia associated with mediastinal germ cell tumor identifies MEK inhibitor as an active clinical agent

Abstract Background Hematologic malignancies arising in the setting of established germ cell tumors have been previously described and have a dismal prognosis. Identification of targetable mutations and pathway dysregulation through massively parallel sequencing and functional assays provides new approaches to disease management. Case Presentation Herein, we report the case of a 23-year-old male who was diagnosed with a mediastinal germ cell tumor and subsequent acute myeloid leukemia. A shared clonal origin was demonstrated through identification of identical NRAS and TP53 somatic mutations in both malignancies. The patient’s leukemia was refractory to standard therapies with short interval relapse. Functional assays demonstrated the patient’s blasts to be sensitive to the mitogen-activated protein kinase kinase (MEK) inhibitor trametinib, correlating with the activating NRAS mutation. The patient experienced a sustained partial remission while on trametinib therapy but ultimately suffered relapse of the germ cell tumor. The leukemic clone remained stable and sensitive to trametinib at that time. Conclusions This case highlights the potential power of combining genetic sequencing and in vitro functional assays with targeted therapies in the treatment of rare diseases

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Supplemental information on the methods of the next-generation sequencing panel at OHSU used for analysis. (DOC 29.5 kb

Additional file 1: of Functional and genetic screening of acute myeloid leukemia associated with mediastinal germ cell tumor identifies MEK inhibitor as an active clinical agent

https://figshare.com/articles/Additional_file_2_Table_S1_of_Functional_and_genetic_screening_of_acute_myeloid_leukemia_associated_with_mediastinal_germ_cell_tumor_identifies_MEK_inhibitor_as_an_active_clinical_agent/4370084

Functional and genetic screening of acute myeloid leukemia associated with mediastinal germ cell tumor identifies MEK inhibitor as an active clinical agent

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